Melissa Verleg, 34, says her life changed dramatically when she started taking Orkambi.

(CHARLOTTE HELSTON / iNFOnews.ca)

September 22, 2017 - 6:30 PM

VERNON - The manufacturer of a life-changing drug for cystic fibrosis patients like Vernon’s Melissa Verleg says it has the evidence to prove the medicine works, but Canada’s drug review agency won’t look at it.

Yesterday, Health Minister Adrian Dix said the Common Drug Review recommended Orkambi not be eligible for funding due to cost and efficacy. That’s left Verleg, and others suffering from the debilitating genetic disease, left hanging because neither health insurers nor FAIR Pharmacare will fully cover the cost — about $255,000 a year per patient.

Vertex Pharmaceuticals spokesperson Megan Goulart says the drug works well in patients with a specific type of cystic fibrosis and they have the research to back it up.

“We share (Verleg’s) and the whole cystic fibrosis community’s frustration with the failure of the Canadian government to provide access to Orkambi,” Goulart says.

She says Health Canada approved the drug for use in January 2016, and Orkambi then went to the Common Drug Review to determine whether it should be eligible for public reimbursement — which it wasn’t.

At the time of the review, Goulart says Vertex submitted the results of two six-month studies, in which half the patients were given a placebo, and the other half received Orkambi. After the six-month trial, Goulart says all patients began receiving Orkambi, and the long-term effects continued to be documented.

She says Vertex wants to resubmit the new data to the Common Drug Review, but they won’t take it. She says that’s because the long-term study involves 100 per cent of participants taking Orkambi, and the review process only accepts data from placebo-based studies.

Goulart says because patients are seeing benefits from the drug, Vertex won’t be conducting another placebo-based study.

“Once you know a medicine works, you can’t not give it. It’s not ethical to withhold it from patients,” Goulart says.

In terms of efficacy, she says Orkambi works on people with a specific type of cystic fibrosis. Another product made by Vertex focusses on a different type of the disease, and between the two, positive results are seen in about one third of patients. For those it does work on, the effects are dramatic, Goulart says.

“For (people like Melissa) it’s had a significant impact on her life,” Goulart says.

She’s says it’s unfortunate that public funding for the drug is mired in procedural hold-ups, leaving patients hanging.

“We are at a road block,” Goulart says. “We really are trying hard to work with the provinces. The national recommendation is not binding — the provincial governments, whether together or individually, can decide if they want to negotiate with us directly.”

iNFOnews.ca contacted the Ministry of Health and asked specifically if that is true, and if the Province of B.C. would consider opening negotiations directly with Vertex. We have not yet received a response.

Goulart also defended the high price of the drug, stating that billions of dollars were spent creating Orkambi and billions more — about 80 per cent of the company's revenues — will be invested into new research for cystic fibrosis medications.

“It’s about reinvesting so we can continue our progress to help more people with this disease,” she says.

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